Apellis Completes Enrollment in Two Phase 3 Studies of Pegcetacoplan in Patients with Geographic Atrophy (GA)

Apellis Pharmaceuticals announced that it completed enrollment in the phase 3 DERBY and OAKS studies investigating intravitreal pegcetacoplan (APL-2), a targeted C3 therapy, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

GA causes blindness and is a progressive complement-driven eye disease that affects approximately 5 million people globally. There are no approved therapies for GA.

“Completing enrollment in the phase 3 DERBY and OAKS studies is a critical milestone that moves us one step closer to bringing the first treatment for geographic atrophy to patients. We are extremely grateful to all patients participating in these groundbreaking studies,” Federico Grossi, MD, PhD, Chief Medical Officer of Apellis, said in a company news release. “People living with GA need a treatment and we believe targeting the complement system at C3 with pegcetacoplan has the potential to control the excessive complement activation that drives the irreversible growth of GA lesions that cause blindness. We look forward to seeing topline results in the third quarter of 2021.”

A total of 1,259 patients are enrolled in DERBY and OAKS, which are pivotal randomized phase 3 studies designed to compare the efficacy and safety of intravitreal pegcetacoplan with sham treatment in patients with GA secondary to AMD. The primary objective of the studies is to evaluate the reduction in growth of GA lesion size, measured by fundus autofluorescence (FAF) at month 12 compared to baseline.

“Geographic atrophy leads to irreversible vision loss, a decline in the quality of life, and remains a major unmet need for our patients. Achieving this critical milestone reflects the importance of bringing this targeted C3 therapy to our GA patients. I want to thank my co-investigators for completing the enrollment of DERBY and OAKS, especially during the ongoing pandemic. I look forward to seeing the results next year with the hope that pegcetacoplan will enable clinicians to treat this debilitating disease,” said Jeffrey S. Heier, MD, Principal Investigator of the DERBY study and Co-President & Medical Director, Director of Retinal Research, Ophthalmic Consultants of Boston.

In July 2018, the FDA granted pegcetacoplan Fast Track Designation for the treatment of GA. The phase 3 DERBY and OAKS studies were initiated based on positive results from the phase 2 FILLY study, which showed that monthly pegcetacoplan treatment resulted in a 29% (P=0.008) reduction in the rate of GA lesion growth compared to sham injections at 12 months.

For more information about the DERBY and OAKS studies, visit www.clinicaltrials.gov: (NCT03525600) and (NCT03525613).